Economic Evaluation of Interventions for Children with Neurodevelopmental Disorders: Practical Guidance

Neurodevelopmental disorders are lifelong conditions with onset in early childhood that impair cognitive, social, behavioural and physical functioning. Their consequences extend beyond healthcare, shaping education, social and community services, and family life. Economic evaluations can guide payers by quantifying the value for money of interventions, but standard methods require adaptation for this context. This paper offers practical guidance for pharmacoeconomic evaluations of interventions for children with neurodevelopmental disorders, synthesising methodological literature and applied experience to articulate core challenges, recommend solutions and highlight emerging opportunities. Evaluations must address five inter-related issues: defining and justifying an analytic perspective that captures multi-sectoral costs and benefits; identifying and valuing direct, indirect and intangible costs across health, education and social domains; selecting outcomes sensitive to changes in child functioning and health-related quality of life that can be economically valued; incorporating spillover effects for caregivers and families; and modelling long-term developmental trajectories amid data scarcity and uncertainty. We recommend adopting a societal perspective; applying comprehensive context-appropriate costing; combining generic and condition-specific outcome measures; explicitly including caregiver and family impacts; integrating equity analyses; and using long time horizons supported by robust modelling such as microsimulation or agent-based approaches. Promising directions include capability-based frameworks and digital biomarkers to enable real-world measurement of function and quality of life. Tailored pharmacoeconomic methods are essential for credible policy-relevant evaluations of neurodevelopmental disorder interventions. Transparent reporting and alignment with international standards will improve comparability, support uptake by decision makers, and promote equitable resource allocation for children and families affected by neurodevelopmental disorders.